5 Participants Needed

ELAPRASE + Prophylactic Therapy for Hunter Syndrome

Recruiting at 10 trial locations
TC
Overseen ByTakeda Contact
Age: < 18
Sex: Male
Trial Phase: Phase 4
Sponsor: Takeda
Must be taking: Elaprase
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 2 JurisdictionsThis treatment is already approved in other countries

What You Need to Know Before You Apply

What is the purpose of this trial?

The main aim of this study is to evaluate the ability of a prophylactic immune tolerizing regimen (ITR) to prevent or reduce the development of high titer anti-idursulfase antibodies in treatment-naïve participants with Hunter syndrome.In this open label, single arm study, all participants will receive ELAPRASE treatment and a prophylactic ITR.Participants will be treated with ELAPRASE for up to 104 weeks. The prophylactic ITR will start 1 day prior to the start of ELAPRASE. The prophylactic ITR will consist of a 5-week cycle of: Rituximab (intravenously \[IV\], weekly for 4 weeks); Methotrexate (oral, 3 times per week for 5 weeks) and intravenous immunoglobulin (IVIG) (IV, every 4 weeks of the cycle).Following the completion of 1 cycle, an assessment will be made at Month 6, 12, and 18 regarding the need for administering another 5-week cycle of the ITR.Participants will be in the study for approximately 112 weeks (including 6 weeks for screening, up to 104 weeks for treatment, and 2 weeks for follow-up).

Will I have to stop taking my current medications?

The trial protocol does not specify if you must stop taking your current medications, but it excludes participants who are currently using any medication that could affect the study. It's best to discuss your current medications with the study team to see if they might impact your eligibility.

Is the treatment with ELAPRASE (idursulfase) generally safe for humans?

ELAPRASE (idursulfase) is generally well tolerated in humans, although some people may experience infusion reactions, which are side effects that occur during or after the drug is given. Serious allergic reactions can happen, and up to 50% of patients may develop antibodies against the treatment, but these are not common.12345

How is the drug Elaprase unique in treating Hunter syndrome?

Elaprase is unique because it is the first enzyme replacement therapy specifically approved for Hunter syndrome, addressing the root cause by replacing the missing enzyme iduronate-2-sulfatase. This treatment helps improve symptoms like walking distance and lung function, and reduces organ size and harmful substances in the body.46789

What data supports the effectiveness of the drug ELAPRASE for Hunter Syndrome?

Research shows that ELAPRASE (idursulfase) helps people with Hunter syndrome by increasing their ability to walk, improving lung function, and reducing the size of organs and certain substances in the urine. It is generally well tolerated, although some people may experience reactions during infusions.45679

Who Is on the Research Team?

SD

Study Director

Principal Investigator

Takeda

Are You a Good Fit for This Trial?

This trial is for treatment-naïve boys under 6 with Hunter syndrome, having specific enzyme deficiencies and gene mutations. They must not have used ELAPRASE or certain other treatments recently and should be able to follow the study protocol without any medical conditions that could interfere.

Inclusion Criteria

I have been diagnosed with MPS II.
Your enzyme activity levels for certain substances in your body are within the normal range.
You do not have antibodies against the drug idursulfase in your blood.
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Exclusion Criteria

Within 30 days prior to the first dose of investigational product, the participant has been enrolled in a clinical study that may impact this study
I am not on any medication that could interfere with the study.
Participant has received treatment with any investigational drug within the 30 days prior to study entry
See 7 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

6 weeks

Treatment

Participants receive ELAPRASE treatment and a prophylactic immune tolerizing regimen (ITR) for up to 104 weeks. The ITR includes Rituximab, Methotrexate, and IVIG in a 5-week cycle.

104 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

2 weeks

What Are the Treatments Tested in This Trial?

Interventions

  • ELAPRASE
  • Intravenous Immunoglobulin (IVIG)
  • Methotrexate
  • Rituximab
Trial Overview The study tests if a preventive immune regimen can stop or lessen high titer anti-idursulfase antibodies in patients taking ELAPRASE. It includes Rituximab, Methotrexate, IVIG alongside ELAPRASE over up to 104 weeks with assessments every six months.
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: ITR + ELAPRASEExperimental Treatment4 Interventions

ELAPRASE is already approved in United States for the following indications:

🇺🇸
Approved in United States as ELAPRASE for:

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Who Is Running the Clinical Trial?

Takeda

Lead Sponsor

Trials
1,255
Recruited
4,219,000+
Dr. Naoyoshi Hirota profile image

Dr. Naoyoshi Hirota

Takeda

Chief Medical Officer since 2020

MD from University of Tokyo

Christophe Weber profile image

Christophe Weber

Takeda

Chief Executive Officer since 2015

PhD in Molecular Biology from Université de Montpellier

Takeda Development Center Americas, Inc.

Industry Sponsor

Trials
58
Recruited
10,800+

Published Research Related to This Trial

Enzyme replacement therapy (ERT) with idursulfase for Hunter syndrome has shown positive effects in patients of all ages, including improvements in weight, height, and a significant reduction in glycosaminoglycan (GAG) levels, which are associated with the disease.
The therapy led to notable clinical improvements, such as recovery from carpal tunnel syndrome and decreased organomegaly, particularly in one patient who experienced an 86% reduction in GAGs, highlighting the efficacy of ERT in managing symptoms and preventing disease progression.
Clinical response to long term enzyme replacement treatment in children, adolescent and adult patients with Hunter syndrome.Dalmau Serra, J., Vitoria Miñana, I., Calderón Fernández, R., et al.[2015]
The study developed specific polyclonal antibodies against human iduronate 2-sulphate sulphatase (IDS), which can be used to detect and quantify IDS in fermentation extracts from Pichia pastoris and Escherichia coli, crucial for enzyme replacement therapy in Hunter syndrome.
These antibodies are effective in distinguishing IDS from other proteins, allowing for their application in both an ELISA system for quantification and in affinity chromatography for purification, enhancing the potential for developing effective treatments for Hunter syndrome.
[Production of polyclonal antibodies to protein iduronate-2-sulphate sulphatase (IDS) and development of a detection system for human recombinant IDS].Peña, O., Sosa, A., Echeverri, O., et al.[2007]
In a study of 124 patients with Hunter syndrome who started enzyme replacement therapy with idursulfase before the age of 6, treatment was found to be safe, with no new safety concerns identified, despite 26.6% experiencing infusion-related reactions.
The therapy demonstrated effectiveness, as evidenced by a significant reduction in urine glycosaminoglycan levels and liver size after at least 6 months of treatment, indicating clinical benefits for young patients.
Idursulfase treatment of Hunter syndrome in children younger than 6 years: results from the Hunter Outcome Survey.Muenzer, J., Beck, M., Giugliani, R., et al.[2022]

Citations

Clinical response to long term enzyme replacement treatment in children, adolescent and adult patients with Hunter syndrome. [2015]
[Production of polyclonal antibodies to protein iduronate-2-sulphate sulphatase (IDS) and development of a detection system for human recombinant IDS]. [2007]
Idursulfase treatment of Hunter syndrome in children younger than 6 years: results from the Hunter Outcome Survey. [2022]
Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome. [2022]
Idursulfase in Hunter syndrome treatment. [2017]
First experience of enzyme replacement therapy with idursulfase in Spanish patients with Hunter syndrome under 5 years of age: case observations from the Hunter Outcome Survey (HOS). [2022]
A multicenter, open-label study evaluating safety and clinical outcomes in children (1.4-7.5 years) with Hunter syndrome receiving idursulfase enzyme replacement therapy. [2022]
Case report of treatment experience with idursulfase beta (Hunterase) in an adolescent patient with MPS II. [2022]
Impact of enzyme replacement therapy on linear growth in Korean patients with mucopolysaccharidosis type II (Hunter syndrome). [2021]
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